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Year Number of Results
2020 1
2021 5
2022 4
2023 1
2024 1

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11 results

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Page 1
Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia.
Moore OM, Aguilar-Sanchez Y, Lahiri SK, Hulsurkar MM, Alberto Navarro-Garcia J, Word TA, Keefe JA, Barazi D, Munivez EM, Moore CT, Parthasarathy V, Davidson J, Lagor WR, Park SH, Bao G, Miyake CY, Wehrens XHT. Moore OM, et al. J Cardiovasc Aging. 2024 Jan;4(1):8. doi: 10.20517/jca.2023.42. Epub 2024 Jan 4. J Cardiovasc Aging. 2024. PMID: 38464671
In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.
De Giorgi M, Park SH, Castoreno A, Cao M, Hurley A, Saxena L, Chuecos MA, Walkey CJ, Doerfler AM, Furgurson MN, Ljungberg MC, Patel KR, Hyde S, Chickering T, Lefebvre S, Wassarman K, Miller P, Qin J, Schlegel MK, Zlatev I, Li RG, Kim J, Martin JF, Bissig KD, Jadhav V, Bao G, Lagor WR. De Giorgi M, et al. bioRxiv [Preprint]. 2023 Jul 29:2023.07.26.550728. doi: 10.1101/2023.07.26.550728. bioRxiv. 2023. PMID: 37546995 Free PMC article. Preprint.
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study.
Doerfler AM, Park SH, Assini JM, Youssef A, Saxena L, Yaseen AB, De Giorgi M, Chuecos M, Hurley AE, Li A, Marcovina SM, Bao G, Boffa MB, Koschinsky ML, Lagor WR. Doerfler AM, et al. Mol Ther Methods Clin Dev. 2022 Oct 13;27:337-351. doi: 10.1016/j.omtm.2022.10.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36381302 Free PMC article.
In vivo genome editing at the albumin locus to treat methylmalonic acidemia.
Schneller JL, Lee CM, Venturoni LE, Chandler RJ, Li A, Myung S, Cradick TJ, Hurley AE, Lagor WR, Bao G, Venditti CP. Schneller JL, et al. Mol Ther Methods Clin Dev. 2021 Nov 11;23:619-632. doi: 10.1016/j.omtm.2021.11.004. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34901307 Free PMC article.
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
CRISPR/Cas9 gene editing for curing sickle cell disease.
Park SH, Bao G. Park SH, et al. Transfus Apher Sci. 2021 Feb;60(1):103060. doi: 10.1016/j.transci.2021.103060. Epub 2021 Jan 10. Transfus Apher Sci. 2021. PMID: 33455878 Free PMC article. Review.
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